Outcome Study of Conventional Steroids Vs. Steroids Combined with Mycopehnolate in Newly Diagnosed Immune Thrombocytopenia Purpura.

NCT06288932 | Completed Phase 3 DMC

• 1 other identifier: NIBD/IRB-263/08-2023
• Study Type: interventional
• Target: 118
• Locations: 1 country
• Sites: 1

Brief Summary

Immune thrombocytopenia Purpura (ITP) is an autoimmune condition delineated by humoral as well as cell mediated immune response against thrombocyte surface proteins GPIIb/IIIa receptors, affecting primary homeostasis leading to mucocutaneous bleeding.ITP is characterized by platelet count \<100 x 109/L. The conventional line of treatment for newly diagnosed ITP is steroids but significant disadvantages have been associated with long term use and a high risk of relapse when reducing the dose. The addition of MMF to the first line treatment of ITP resulted in substantial response and a lower risk of refractory ITP with decreased financial burden and improved outcome.

Conditions

Immune Thrombocytopenia Purpura

Keywords

ITP; MMF

Outcome Measures

Primary Outcomes (1)

• Platelet count monitoring

  Platelet count improvement, as assessed through regular monitoring and according to response criteria: The response criteria to the treatment described as follow: 1. Complete response: Platelet count ≥ 100 x 10\^ 9 /L 2. Partial response: Platelet count 30≥ 100 x 10 \^9 /L 3. No response: Platelet count 30 x 10\^9 /

  assessed after 3 months and 6 months of treatment.

Secondary Outcomes (2)

• Assessment of Health-Related Quality of Life questionnaire

  12 months

• Incidence of adverse events

  12 months

Study Arms (2)

Mycophenolate Mofetil (MMF) Treatment

EXPERIMENTAL

Patients in this arm will receive Mycophenolate Mofetil (MMF) at a starting dose of 500mg twice daily along with Prednisolone. The pediatric dose of MMF will be calculated by 30mg/kg/day. Dose adjustment will be based on treatment response, with potential increases to 750mg twice daily or 1g twice daily. MMF will be administered for a specific duration, as determined by the study design.

Drug: Mycophenolate Mofetil (MMF) Treatment

Standard Treatment Control

NO INTERVENTION

Patients in this arm will receive the standard treatment for ITP, which may include corticosteroids (prednisolone) commonly used for ITP management.The treatment regimen will be determined based on current standard guidelines and clinical practice.

Interventions

Mycophenolate Mofetil (MMF) Treatment DRUG

The first-line treatment of immune thrombocytopenia purpura with Mycophenolate Mofetil produced a significant response, a lowered likelihood of refractory ITP, a better outcome, and less financial burden.

Also known as: Suprimun

Mycophenolate Mofetil (MMF) Treatment

Eligibility Criteria

• Age: 5 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

Inclusion Requirements: • Age: 5-60 years • Number of plates: \<30x109 Exclusion Standards: Pregnancy; lactation; hepatitis B and C; HIV-reactive individuals; and medication allergies

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Institute of Blood and Marrow Transplant (NIBMT), Pakistan: lead

Study Sites (1)

National Institute of Blood Diseases and Bone Marrow Transplantation

Karachi, Sindh, 75300, Pakistan

Location

MeSH Terms

Interventions

Mycophenolic Acid; Therapeutics

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids

Study Officials

• Rukh-e- Zainub, Pharm-D,Mphil(enrolled)

  National Institute of Blood Diseases and Bone Marrow Transplantation

  PRINCIPAL INVESTIGATOR

• Shafaq Abdul Samad, MBBS,FCPS

  National Institute of Blood Diseases and Bone Marrow Transplantation

  STUDY DIRECTOR

• Dr.Iyad Naeem, Ph.D

  KARACHI UNIVERSITY

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Model Details: The study will comprise the newly diagnosed patients of Immune thrombocytopenia purpura aged 5-60 year. Patients will with a 1:1 ratio among the two group.

Study Record Dates

• First Submitted: February 16, 2024
• First Posted: March 1, 2024
• Study Start: August 18, 2023
• Primary Completion: February 18, 2024
• Study Completion: August 18, 2024
• Last Updated: November 6, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

PA (1)